FDA Grants Accelerated Approval to Tisagenlecleucel for R/R Follicular Lymphoma


Results from the phase 2 ELARA trial supported an accelerated approval from the FDA for tisagenlecleucel for patients with relapsed/refractory follicular lymphoma.

The FDA has granted accelerated approval to tisagenlecleucel (tisa-cel; Kymriah) for adult patients with relapsed or refractory follicular lymphoma following 2 or more lines of systemic therapy, according to a press release from Novartis.

The approval is based on the phase 2 ELARA trial (NCT03568461), a single-arm open-label study that assessed the tisagenlecleucel as a single-infusion in patients with relapsed/refractory follicular lymphoma. Overall, 86% of patients experienced a response following treatment with tisa-cel, of whom 68% achieved a complete response. Additionally, 85% of patients who achieved a complete response continued to respond 12 months after their initial response.

“We are proud of today’s FDA approval of a third indication for [tisa-cel]. We hope this treatment option that has the potential for long-lasting results may help break the unrelenting cycle of treatment for patients with follicular lymphoma,” Victor Bulto, president of Novartis Innovative Medicines in the United States, said in the press release. “We are on a mission to build on our pioneering work in cell therapy and continue to innovate for patient impact.”

A total of 90 patients were evaluable for efficacy at a median follow-up of 17 months. The primary end point was complete response rate, with key secondary end points including overall response rate, duration of response, progression-free survival, and overall survival.

Safety was evaluable in 97 patients at a median follow-up 21 months. Any-grade cytokine release syndrome was observed in 53% of patients, and there was no grade 3 or higher events. Additionally, 43% of patients experienced any-grade neurologic events, of whom 6% experienced grade 3 or higher events. Additionally, 18% of patients had an infusion in the outpatient setting.

Inclusion criteria required patients to have grade 1, 2, or 3A relapsed or refractory follicular lymphoma, and radiographically measurable disease at screening. Exclusion criteria included evidence of histologic transformation, follicular lymphoma that was grade 3B, prior anti-CD-19 therapy, and prior gene therapy.

Investigators noted that patients with follicular lymphoma may be exposed to a median of 4 lines of prior therapy with an upper range of 13 lines. The European Commission has previously approved this treatment in early May 2022.

“The approval of [tisa-cel] offers patients with relapsed or refractory follicular lymphoma a new treatment option and new hope for improving patient outcomes,” Meghan Gutierrez, chief executive officer at the Lymphoma Research Foundation, concluded. “Having this single infusion treatment option helps to transform the way healthcare providers approach this type of blood cancer and we commend those who have contributed to the acceleration of scientific research for the benefit of patients.”


FDA approves Novartis Kymriah CAR-T cell therapy for adult patients with relapsed or refractory follicular lymphoma. News Release. Novartis. May 28, 2022. Accessed May 31, 2022. https://bit.ly/390omqP

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